Novo Nordisk bags Prothena's ATTR amyloidosis programme for up to $1.2 billion

Novo Nordisk has entered into a deal to acquire Prothena's experimental anti-amyloid immunotherapy PRX004 as well as its broader transthyretin (ATTR) amyloidosis programme, the companies announced Monday. Under the deal, Novo Nordisk will pay Prothena $100 million in upfront and near-term clinical milestones, with the latter eligible to receive up to $1.2 billion if certain development and sales targets are met.

"With its innovative amyloid-depleting mechanism, PRX004 has the potential to offer a novel treatment option for ATTR cardiomyopathy," remarked Marcus Schindler, head of research and early development at Novo Nordisk.

Prothena has completed a Phase I study with PRX004 in patients with hereditary forms of ATTR, in which the treatment slowed neuropathy progression for all seven evaluable patients at nine months, including improving neuropathy in three of the subjects, and improving cardiac systolic function for all seven (see ViewPoints: Prothena's amyloidosis bet gets bigger). The companies noted that PRX004 was also found to be safe and well tolerated.

Initial focus on ATTR cardiomyopathy

Novo Nordisk said it will initially focus on the clinical development of PRX004 in ATTR cardiomyopathy. Under the agreed terms, Novo Nordisk acquires Prothena's wholly-owned subsidiary and gains full worldwide rights to the intellectual property and related rights of Prothena's ATTR amyloidosis business and pipeline.

Hideki Garren, chief medical officer at Prothena, stated "we are confident that Novo Nordisk will…advance [PRX004] on an expedited timeline." The executive added that "with Novo Nordisk's commitment to further develop PRX004 in ATTR cardiomyopathy, [we] will continue to focus on our mission to advance our…portfolio designed to address rare peripheral amyloid and neurodegenerative diseases."

Late last month, Intellia Therapeutics and Regeneron Pharmaceuticals reported the first-ever data for in vivo CRISPR genome editing in humans, with positive interim findings of the CRISPR/Cas9-based therapy NTLA-2001 in six patients with ATTR amyloidosis with polyneuropathy who had received a single infusion. For additional analysis, see ViewPoints: Patience pays off at Intellia.

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